Welcome to the muscular dystrophy information page.
Learn about muscular dystrophy, its types, clinical trials, and more.
Welcome to the muscular dystrophy information page.
Muscular dystrophy is a group of genetic disorders causing progressive muscle weakness and degeneration. These conditions occur due to mutations in genes that affect proteins needed for proper muscle function, particularly dystrophin.
DMD is the most severe and common type of muscular dystrophy in children. It primarily affects boys (X-linked inheritance) and causes progressive muscle weakness, difficulty walking, and eventually affects heart and respiratory function.
Clinical trials for muscular dystrophy focus on gene therapy, exon-skipping drugs, stem cell therapy, and anti-inflammatory treatments. These trials aim to slow disease progression and improve quality of life for patients.
Current research directions include gene therapy to deliver functioning copies of the dystrophin gene, exon-skipping to restore partial protein function, and pharmacological approaches to reduce inflammation and maintain muscle strength.
Clinical trials follow a structured approach moving through phases:
Based on the dataset featured on this page, clinical trials for DMD typically last 7-8 years, with:
The visualizations below display data from clinical trials for Duchenne Muscular Dystrophy, focusing on two key functional assessments: the 6-Minute Walk Test (6MWT) and the North Star Ambulatory Assessment (NSAA).
About the 6-Minute Walk Test (6MWT): This test measures how far a patient can walk in six minutes on a flat, 30-meter track. It evaluates endurance, muscle fatigue, and disease progression. A declining score over time typically indicates disease progression. Healthy children can walk 400-700 meters, while those with advanced DMD may walk less than 200 meters.
About the North Star Ambulatory Assessment (NSAA): This assessment evaluates motor functions like standing up, climbing stairs, and balancing. Scores range from 0 to 34, with higher scores indicating better motor function. The NSAA is particularly sensitive to changes in ambulatory function in children with DMD.
The table below shows raw data from the clinical trials dataset, including patient IDs, ages, and their functional test scores.
| ID | Age | 6MWT | NSAA |
|---|
Clinical trials for muscular dystrophy use various assessments to evaluate treatment efficacy and disease progression: